Positive high-level results from a late-stage study of a drug for those with a rare hereditary nerve disease are expected to lead AstraZeneca to apply for regulatory approval.
The NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed AstraZeneca and Ionis’ eplontersen significantly improved patient-reported quality of life versus an external placebo group.
Ionis is a biotech company based in Carlsbad, north of San Diego, CA. AstraZeneca, based in the United Kingdom, employed 1,500 in Delaware at last report.
ATTRv-PN is a debilitating disease that leads to nerve damage and, without treatment, is generally fatal within a decade. Eplontersen is a medicine designed to reduce the production of TTR protein at its source to treat both hereditary and non-hereditary forms of ATTR.
Teresa Coelho, M.D., a neurologist and neurophysiologist at Hospital Santo António, Centro Hospitalar Universitário do Porto, Portugal and an investigator for the trial, said: “These encouraging data reinforce the safety profile of eplontersen and demonstrate clear evidence of its potential to provide much needed therapeutic benefit to patients living with hereditary transthyretin-mediated amyloid polyneuropathy.”
Mene Pangalos, executive vice president, BioPharmaceuticals R&D at AstraZeneca, said: “Amyloid transthyretin polyneuropathy is a rare and fatal disease that can affect up to 40,000 people worldwide. These promising results show eplontersen has the potential to be a new and much-needed treatment where limited options exist and significant unmet medical need remains.”
Based on the 35-week interim trial results, the companies will seek regulatory approval for eplontersen and plan to file a new drug application with the US Food and Drug Administration in 2022.
As part of a global development and commercialization agreement with Ionis, eplontersen will be jointly developed and commercialized by both companies in the US and will be developed and commercialized in the rest of the world by AstraZeneca (with the exception of Latin America).
Eplontersen was granted Orphan Drug Designation in the US and is also currently being evaluated for amyloid transthyretin cardiomyopathy, condition that leads to progressive heart failure and death within four years from diagnosis.
